by Peter Ciszewski | Dec 8, 2017
The rare disease industry – including patients, researchers, and biotech companies – are concerned that a key financial provision in the Orphan Drug Act of 1983 may soon be removed. These financial incentives have been key driver for biotech companies to pursue...
by Peter Ciszewski | Dec 8, 2017
Spark Therapeutics and Pfizer announced that The New England Journal of Medicine has published interim data from the Phase 1/2 clinical trial of SPK-9001, an investigational gene therapy for hemophilia B. With a cumulative follow-up of 492 weeks’ observation of the...
by Peter Ciszewski | Dec 7, 2017
David Gelly is a patient with Polycythemia Vera (PV). In this interview he discusses some of the resources available to patients with this rare condition, including Voices of MPN. PVs are a group of rare blood cancers called Myeloproliferative Neoplasms (MPNs). MPNs...
by Peter Ciszewski | Dec 6, 2017
David G. Birch, PhD, Chief Scientific and Executive Officer and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, Retina Foundation of the Southwest discusses genetic testing and X-linked retinoschisis (XLRS). X-linked juvenile retinoschisis is a...
by Peter Ciszewski | Dec 6, 2017
Sandesh Seth, Executive Chairman of Actinium Pharmaceuticals discusses the potential market for Iomab-B. Iomab-B via the monoclonal antibody BC8, targets CD45, an antigen widely expressed on leukemia and lymphoma cancer cells, B cells and stem cells. BC8 is linked to...
